Agios’ Pyrukynd (mitapivat) Receives EMA's CHMP Positive Opinion Recommending the Marketing Authorization for the Treatment of PK Deficiency
Shots:
- The CHMP opinion was based on the P-III (ACTIVATE) & (ACTIVATE-T) studies evaluating Pyrukynd in patients with PK deficiency. The EC’s decision was expected within 67 Days
- Both the trials met their 1EPs i.e., patients achieved a hemoglobin response (40% vs 0%) over PBO in (ACTIVATE) trial & significant improvements for all pre-specified 2EPs incl. markers of hemolysis & ineffective erythropoiesis. In (ACTIVATE-T) trial, 37% achieved a ≥33% reduction in transfusion burden @24wk. fixed dose period, 22% were transfusion-free
- If EC approved Pyrukynd, it will be 1st approved disease-modifying therapy for EU patients. The company also continues to advance the therapy in the 2 P-III trials for thalassemia & P-II/III (RISE UP) study in SCD
Ref: Globenewswire| Image: Agios
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